FDA de­rails Acor­da's mar­ket­ing pitch, cre­at­ing a cri­sis as flag­ship drug founders

Acor­da just en­tered a world of hurt.

With its patent pro­tec­tion crum­bling around its main­stay drug, the FDA has re­fused to file its ap­pli­ca­tion for CVT-301, the res­cue pro­gram that was des­ig­nat­ed to take the lead drug’s place. The RTF cre­ates an em­bar­rass­ing and per­haps crip­pling prob­lem for the biotech, which is fight­ing a last-ditch bat­tle to main­tain patent pro­tec­tion on its one big drug, Ampyra.

Ron Co­hen

Ac­cord­ing to Acor­da, the FDA says the com­pa­ny’s mar­ket­ing ap­pli­ca­tion, rushed to reg­u­la­tors as the com­pa­ny re­struc­tured and down­sized, is in­com­plete. Reg­u­la­tors said they were un­able to do a prop­er re­view as they don’t know what date Acor­da’s man­u­fac­tur­ing cen­ter will be ready for in­spec­tion and have a “ques­tion re­gard­ing the sub­mis­sion of the drug mas­ter pro­duc­tion record.”

Acor­da re­port­ed that the FDA al­so re­quest­ed ad­di­tion­al in­for­ma­tion at re­sub­mis­sion, which was not part of the ba­sis for the RTF.

Its shares quick­ly plunged 26% on the news.

Nor­mal­ly, a de­lay like this would be seen as a sticky prob­lem, but one that could po­ten­tial­ly be re­solved with­out huge de­lays. The prob­lem for Acor­da, though, is that the com­pa­ny has vir­tu­al­ly no lee­way for a sna­fu like this. Not on­ly does it leave Acor­da’s ex­ec­u­tive team with a black eye on ex­e­cu­tion, it cre­ates se­ri­ous doubts about its abil­i­ty to quick­ly and ef­fi­cient­ly line up a re­place­ment drug for its sales team by next sum­mer, as the lit­i­ga­tion over its patents for Ampyra comes to a head.

Leerink’s Paul Mat­teis found the whole thing puz­zling and dis­con­cert­ing, con­sid­er­ing the like­li­hood of a loom­ing ap­proval. And he still has ques­tions about what is go­ing on at Acor­da.

It seems like the best case sce­nario would be a re-sub­mis­sion around the end of this year, and its en­cour­ag­ing that FDA did not make any re­quests re­gard­ing the ef­fi­ca­cy or safe­ty da­ta. Nonethe­less, even af­ter speak­ing with the com­pa­ny, the RTF is mys­te­ri­ous to us, and we ex­pect the stock to be un­der pres­sure un­til opac­i­ty is lift­ed.

As I re­port­ed in June, while re­view­ing the late-stage da­ta sub­mit­ted to the FDA on the in­haled lev­odopa, any set­back on CVT-301 would be a cat­a­stro­phe for Acor­da.

Acor­da shift­ed to sur­vival mode in ear­ly April af­ter a US dis­trict court tossed four key patents on its flag­ship drug Ampyra, leav­ing it with one to stand on in­to next year. The com­pa­ny chopped 20% of its staff as it scram­bles to re­struc­ture while gam­bling that it can field new drugs in short or­der.

Now Acor­da is left to des­per­ate­ly seek a quick meet­ing with reg­u­la­tors to clear up any of these ques­tions. But the FDA is not known for scram­bling a re­sponse in any of these sit­u­a­tions, leav­ing Acor­da’s time­line in the air just when it most needs cer­tain­ty. Just weeks ago one of its biggest in­vestors pushed CEO Ron Co­hen to auc­tion the com­pa­ny off rather than risk the con­se­quences of fail­ing at its high wire act. The com­pa­ny pushed back, but a sale may come back on the ta­ble now whether or not it likes the idea.

“We will work with the FDA as quick­ly as pos­si­ble to ad­dress the open is­sues and to clar­i­fy the path to suc­cess­ful­ly re-file our ap­pli­ca­tion,” said Co­hen in a state­ment. “We re­main con­fi­dent in IN­BRI­JA’s da­ta pack­age and its promise as an im­por­tant new ther­a­py for peo­ple with Parkin­son’s dis­ease. We see tremen­dous long-term val­ue in its sol­id clin­i­cal pro­file, sig­nif­i­cant com­mer­cial op­por­tu­ni­ty and strong IP, and we re­main fo­cused on work­ing to bring pa­tients this im­por­tant new ther­a­py.”

Martin Shkreli [via Getty]

Pris­on­er #87850-053 does not get to add drug de­vel­op­er to his list of cred­its

Just days after Retrophin shed its last ties to founder Martin Shkreli, the biotech is reporting that the lead drug he co-invented flopped in a pivotal trial. Fosmetpantotenate flunked both the primary and key secondary endpoints in a placebo-controlled trial for a rare disease called pantothenate kinase-associated neurodegeneration, or PKAN.

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We­bi­nar: Re­al World End­points — the brave new world com­ing in build­ing fran­chise ther­a­pies

Several biopharma companies have been working on expanding drug labels through the use of real world endpoints, combing through the data to find evidence of a drug’s efficacy for particular indications. But we’ve just begun. Real World Evidence is becoming an important part of every clinical development plan, in the soup-through-nuts approach used in building franchises.

I’ve recruited a panel of 3 top experts in the field — the first in a series of premium webinars — to look at the practical realities governing what can be done today, and where this is headed over the next few years, at the prodding of the FDA.

ZHEN SU — Merck Serono’s Senior Vice President and Global Head of Oncology
ELLIOTT LEVY — Amgen’s Senior Vice President of Global Development
CHRIS BOSHOFF — Pfizer Oncology’s Chief Development Officer

A premium subscription to Endpoints News is required to attend this webinar. Please upgrade to either an Insider or Enterprise plan for access. Already have Endpoints Premium? Please sign-in below. You can contact our Subscriptions team at help@endpointsnews.com with any issues.

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Hal Barron. GSK

GSK's Hal Bar­ron her­alds their sec­ond pos­i­tive piv­otal for cru­cial an­ti-BC­MA ther­a­py, point­ing to a push for quick OKs in a crowd­ed field

Hal Barron has his second positive round of Phase III data in hand for his anti-BCMA antibody drug conjugate belantamab mafodotin (GSK2857916). And GSK’s research chief says the data paves the way for their drive in search of an FDA approval for treating multiple myeloma.

It’s hard to overestimate the importance of this drug for GSK, a cornerstone of Barron’s campaign to make a dramatic impact on the oncology market and provide some long-lost excitement for the pharma giant’s pipeline. They’re putting this BCMA program at the front of that charge — looking to lead a host of rivals all aimed at the same target.

We don’t know what the data are yet, but DREAMM-2 falls on the heels of a promising set of data delivered 5 months ago for DREAMM-1. There investigators noted that complete responses among treatment-resistant patients rose to 15% in the extra year’s worth of data to look over, with a median progression-free survival rate of 12 months, up from 7.9 months reported earlier. The median duration of response was 14.3 months.

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Brian Kaspar. AveXis via Twitter

AveX­is sci­en­tif­ic founder fires back at No­var­tis CEO Vas Narasimhan, 'cat­e­gor­i­cal­ly de­nies any wrong­do­ing'

Brian Kaspar’s head was among the first to roll at Novartis after company execs became aware of the fact that manipulated data had been included in its application for Zolgensma, now the world’s most expensive therapy.

But in his first public response, the scientific founder at AveXis — acquired by Novartis for $8.7 billion — is firing back. And he says that not only was he not involved in any wrongdoing, he’s ready to defend his name as needed.

I reached out to Brian Kaspar after Novartis put out word that he and his brother Allen had been axed in mid-May, two months after the company became aware of the allegations related to manipulated data. His response came back through his attorneys.

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Why would Am­gen want to buy Alex­ion? An­a­lysts call hot­ly ru­mored takeover un­like­ly, but seize the mo­ment

A rumor that Amgen is closing in on buyout deal for Alexion has sparked a guessing game on just what kind of M&A strategy Amgen is pursuing and how much Alexion is worth.

Mizuho analyst Salim Syed first lent credence to the report out of the Spanish news outlet Intereconomía, which said Amgen is bidding as much as $200 per share. While the source may be questionable, “the concept of this happening doesn’t sound too crazy to me,” he wrote.

FDA asks why No­var­tis took two months to launch for­mal in­ter­nal probe, af­ter AveX­is flagged da­ta ma­nip­u­la­tion

And the plot thickens. Novartis $NVS officials are reportedly now scrambling to explain to the FDA why it took them two months to open an internal investigation into data discrepancies for their $2.1 million gene-therapy for spinal muscular dystrophy — the world’s most expensive drug.

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Build­ing on suc­cess­ful PD-1 pact, Eli Lil­ly li­cens­es di­a­betes drug to Chi­nese part­ners at In­novent

Eli Lilly is expanding its partnership with China’s Innovent in a deal involving a diabetes drug sitting in its Phase I reserves.

The two companies had jointly developed one of China’s first homegrown PD-1 agents, scoring an approval for Tyvyt (sintilimab) late last year for relapsed/refractory classical Hodgkin’s lymphoma. This time around, Lilly is out-licensing a piece of its diabetes pipeline, a leading franchise that has historically produced the top-selling Trulicity and Humalog.

UP­DAT­ED: An em­bold­ened As­traZeneca splurges $95M on a pri­or­i­ty re­view vouch­er. Where do they need the FDA to hus­tle up?

AstraZeneca is in a hurry.

We learned this morning that the pharma giant — not known as a big spender, until recently — forked over $95 million to get its hands on a priority review voucher from Sobi, otherwise known as Swedish Orphan Biovitrum.

That marks another step down on price for a PRV, which allows the holder to slash 4 months off of any FDA review time.

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Bob Smith, Pfizer

Pfiz­er is mak­ing a $500M state­ment to­day: Here’s how you be­come a lead play­er in the boom­ing gene ther­a­py sec­tor

Three years ago, Pfizer anted up $150 million in cash to buy Bamboo Therapeutics in Chapel Hill, NC as it cautiously stuck a toe in the small gene therapy pool of research and development.

Company execs followed up a year later with a $100 million expansion of the manufacturing operations they picked up in that deal for the UNC spinout, which came with $495 million in milestones.

And now they’re really going for it.

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